Petersen-Jones Simon M, Komáromy András M
1 Department of Small Animal Clinical Sciences, Michigan State University , East Lansing, MI 48824.
Hum Gene Ther Clin Dev. 2015 Mar;26(1):15-26. doi: 10.1089/humc.2014.155. Epub 2015 Feb 11.
Spontaneous canine models exist for several inherited retinal dystrophies. This review will summarize the models and indicate where they have been used in translational gene therapy trials. The RPE65 gene therapy trials to treat childhood blindness are a good example of how studies in dogs have contributed to therapy development. Outcomes in human clinical trials are compared and contrasted with the result of the preclinical dog trials.
存在几种遗传性视网膜营养不良的自发性犬类模型。本综述将总结这些模型，并指出它们在转化基因治疗试验中的应用情况。治疗儿童失明的RPE65基因治疗试验是犬类研究如何促进治疗发展的一个很好的例子。将人类临床试验的结果与临床前犬类试验的结果进行比较和对比。
